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Advances in genetic technologies and in the understanding of clinically relevant genetic variation are revolutionising how a whole range of diseases can be diagnosed and treated. This collection of articles offers a glimpse into research that is laying important groundwork for genomic medicine to play a transformative part in patient healthcare. The selected articles explore new approaches to prevent infectious disease, treat cancer and, potentially, cure inherited disorders using knowledge of the genome.
A switch system is developed to control the expression of therapeutic genes, involving the administration of a small-molecule drug to induce splicing-mediated control of mRNA translation.
A custom adenine base editor can edit the variant of the β-globin gene that causes sickle cell disease into a non-pathogenic variant in human and mouse cells, and transplantation of the edited cells rescues sickle cell disease in mice.
Genomic integration of an adeno-associated virus vector in a mouse model of Angelman syndrome unsilences paternal Ube3a and rescues anatomical and behavioural phenotypes, suggesting a pathway towards the treatment of this neurodevelopmental disorder.
A single, low-dose intradermal immunization with lipid-nanoparticle-encapsulated nucleoside-modified mRNA encoding the pre-membrane and envelope glycoproteins of Zika virus protects both mice and rhesus macaques against infection and elicits rapid and long-lasting neutralizing antibody responses.